CN116024266A - 用于治疗由于nd4突变造成的莱伯氏遗传性视神经病变的组合物和方法 - Google Patents

用于治疗由于nd4突变造成的莱伯氏遗传性视神经病变的组合物和方法 Download PDF

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CN116024266A
CN116024266A CN202210938308.5A CN202210938308A CN116024266A CN 116024266 A CN116024266 A CN 116024266A CN 202210938308 A CN202210938308 A CN 202210938308A CN 116024266 A CN116024266 A CN 116024266A
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李斌
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Wuhan Niufusi Biological Technology Co ltd
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Wuhan Niufusi Biological Technology Co ltd
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CN202210938308.5A 2021-08-06 2022-08-05 用于治疗由于nd4突变造成的莱伯氏遗传性视神经病变的组合物和方法 Pending CN116024266A (zh)

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CN202110899561X 2021-08-06
CN202110899561 2021-08-06

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CN116024266A true CN116024266A (zh) 2023-04-28

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CN202210938308.5A Pending CN116024266A (zh) 2021-08-06 2022-08-05 用于治疗由于nd4突变造成的莱伯氏遗传性视神经病变的组合物和方法

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US (1) US20240376164A1 (https=)
EP (1) EP4382601A4 (https=)
JP (1) JP2024529033A (https=)
KR (1) KR20240042489A (https=)
CN (1) CN116024266A (https=)
AR (1) AR126727A1 (https=)
AU (1) AU2022322957A1 (https=)
BR (1) BR112024002305A2 (https=)
CA (1) CA3228344A1 (https=)
MX (1) MX2024001597A (https=)
WO (1) WO2023011632A1 (https=)

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3625214A (en) 1970-05-18 1971-12-07 Alza Corp Drug-delivery device
US4906474A (en) 1983-03-22 1990-03-06 Massachusetts Institute Of Technology Bioerodible polyanhydrides for controlled drug delivery
US4789734A (en) 1985-08-06 1988-12-06 La Jolla Cancer Research Foundation Vitronectin specific cell receptor derived from mammalian mesenchymal tissue
AU610083B2 (en) 1986-08-18 1991-05-16 Clinical Technologies Associates, Inc. Delivery systems for pharmacological agents
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US6024983A (en) 1986-10-24 2000-02-15 Southern Research Institute Composition for delivering bioactive agents for immune response and its preparation
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
DK0678034T3 (da) 1993-01-11 1999-11-08 Dana Farber Cancer Inst Inc Induktion af cytotoksiske T-lymfocytreaktioner
US6207392B1 (en) 1997-11-25 2001-03-27 The Regents Of The University Of California Semiconductor nanocrystal probes for biological applications and process for making and using such probes
CN1206361C (zh) * 1999-09-10 2005-06-15 本元正阳基因技术股份有限公司 系列通用型腺病毒伴随病毒载体的构建及用途
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
CN111068071A (zh) * 2018-10-22 2020-04-28 武汉纽福斯生物科技有限公司 基因治疗Leber遗传学视神经病变
CN113528510A (zh) * 2018-06-29 2021-10-22 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
SG11202101032VA (en) * 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy
EP3908656A4 (en) * 2019-01-07 2022-10-05 California Institute of Technology GAS VESICLE EXPRESSION SYSTEMS, GAS VESICLE CONSTRUCTS AND ASSOCIATED GENETIC CIRCUITS, VECTORS, MAMMALIAN CELLS, HOSTS, COMPOSITIONS, METHODS AND SYSTEMS
CN113025633B (zh) * 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
WO2021138559A1 (en) * 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

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AR126727A1 (es) 2023-11-08
CA3228344A1 (en) 2023-02-09
AU2022322957A1 (en) 2024-03-21
KR20240042489A (ko) 2024-04-02
MX2024001597A (es) 2024-02-15
BR112024002305A2 (pt) 2024-04-30
WO2023011632A1 (zh) 2023-02-09
US20240376164A1 (en) 2024-11-14
EP4382601A4 (en) 2025-07-30
JP2024529033A (ja) 2024-08-01
EP4382601A1 (en) 2024-06-12

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